Next Topics

Hemophilia A

New England CEPAC

The Institute for Clinical and Economic Review (ICER) will develop a report assessing the comparative clinical effectiveness and value of emicizumab (Genentech/Roche), a monoclonal antibody currently under review by the FDA for prevention of bleeding in patients who have hemophilia A with coagulation factor VIII inhibitors. Factor VIII is the clotting protein that is deficient in hemophilia A, and inhibitors interfere with factor replacement therapy. An FDA approval decision is expected in the first quarter of 2018.

Low Back Pain

CTAF, ICER, Washington State

ICER will develop a report assessing the comparative clinical effectiveness and value of certain non-pharmacologic, non-invasive interventions for chronic low back and neck pain. The report will be the subject of a October 19, 2017 meeting of the California Technology Assessment Forum.

Interventions of Interest:

  • Acupuncture
  • Cognitive behavioral therapy
  • Mindfulness
  • Yoga
  • Tai Chi

Date of review: October 2017

Tardive Dyskinesia

New England CEPAC

Tardive dyskinesia is a serious and disabling movement disorder that affects individuals treated with antipsychotic agents for a variety of mental health conditions. Though prevention and treatment primarily focus on stopping or changing the offending antipsychotic agent, for some individuals there are no other effective treatment options, and symptoms can persist after discontinuing or changing antipsychotic therapy.

Interventions of Interest:

  • Valbenazine (IngrezzaTM, Neurocrine Biosciences, Inc)
  • Deutetrabenazine (AustedoTM, Teva)
  • Tetrabenazine (Xenazine®)

Date of Review: December 2017

Voretigene Neparvovec

Midwest CEPAC

ICER will develop a report assessing the comparative clinical effectiveness and value of voretigene neparvovec (Spark Therapeutics), a potentially one-time gene therapy for vision loss associated with biallelic RPE65-mediated retinal disease. Voretigene neparvovec is the first gene therapy to be submitted for approval by the US Food and Drug Administration (FDA).

Date of review: January 2018

CAR-T Therapies


ICER will develop a report on chimeric antigen receptor t-cell (CAR-T) therapies for treatment of B-cell malignancies. ICER’s report is expected to consider the comparative clinical effectiveness and value of tisagenlecleucel (KymriahTM [CTL-019], Novartis) and axicabtagene ciloleucel (KTE-019, Kite Pharma). Tisagenlecleucel was approved by the FDA is August of 2017; an approval decision for axicabtagene ciloleucel is expected in December of 2017, respectively.  If approved, tisagenlecleucel-t is expected to be used in the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia in pediatric and young adult patients. Novartis is also expected to file later this year for a second indication for diffuse large B-cell lymphoma in adults. Axicabtagene ciloleucel is under review for the treatment of relapsed or refractory aggressive non-Hodgkin’s lymphoma in adult patients who are not eligible for autologous stem cell transplant.

Date of Review: March 2018

Cystic Fibrosis

Midwest CEPAC

past Topics

Sep 2017
Midwest CEPAC

Ovarian Cancer

ICER’s report reviews evidence on the comparative clinical effectiveness and value of poly (ADP-ribose) polymerase (PARP) inhibitors for treatment of ovarian cancer.

Interventions of Interest:

  • Rucaparib (Rubraca,®, Clovis Oncology)
  • Niraparib (Zejula®, Tesaro, Inc.)
  • Olaparib (Lynparza®, AstraZeneca)
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Jul 2017
New England CEPAC

Abuse-Deterrent Opioids

Opioids are used to treat cases of acute and chronic pain that arise from a variety of causes, ranging from trauma to palliative care for advanced illness. Every year, 100 million people in the United States suffer from pain, 9-12 million of whom have chronic or persistent pain. Although opioid therapy is an important component of pain management for many patients, the addictive and euphoric properties of these drugs make them liable to misuse, abuse, addiction, and diversion.

In an effort to help tackle the public health crisis of opioid dependence, misuse, and addiction that has emerged over the last decade, drug manufacturers have begun to develop abuse-deterrent formulations of opioid medications (ADFs). ICER will review available evidence on the comparative clinical effectiveness and comparative value of ADFs in order to inform decision-making by patients, clinicians and policy-makers, within the context of multiple efforts being undertaken to combat the opioid crisis. ICER’s review will also analyze the potential of ADFs to reduce the burden of prescription opioid abuse by evaluating their benefits and market penetration based on coverage policies and legislation.

Interventions of Interest:

  • Hysingla® ER (Hydrocodone, Purdue)
  • Vantrela® (Hydrocodone , Teva)
  • Arymo® ER ( Morphine, Egalet)
  • Embeda® (Morphine + naltrexone, Pfizer)
  • MorphabondTM (Morphine extended release, Inspirion Delivery Technologies)
  • OxyContin® TR (Oxycodone, Purdue)
  • Xtampza®ER (Oxycodone, Collegium Pharmaceutical Inc)
  • Targiniq® (Oxycodone + naloxone extended release, Purdue)
  • Troxyca® ER (Oxycodone + naltrexone, Pfizer)
  • RoxyBond® (Oxycodone, Inspirion Delivery Technologies)

Date of Review: July 2017

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Jun 2017


Osteoporosis, the weakening of the bones through loss of bone mineral content and a decrease in bone quality, is a common disease of aging that affects approximately 10 million Americans. Approximately half of women and one quarter of men will suffer at least one fracture due to osteoporosis during their lifetimes. Experts estimate that there are approximately two million osteoporotic fractures each year, which results in $19 billion in related costs. By 2025, these figures are predicted to grow to approximately three million fractures and $25 billion in costs annually as the population of older Americans increases.

Interventions of Interest:

  • Abaloparatide (Radius Health, Inc.)
  • Teriparatide (Forteo®, Eli Lilly and Co.)
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May 2017

Orphan Drug Summit

ICER will host a multi-stakeholder policy summit to discuss methods for assessing the value of new drugs for rare conditions. The summit will include discussion of how these methods can be applied to recommend fair prices that reflect the value of orphan drugs to patients and the health system to allow for broader insurance coverage for innovative new treatments.

More information about the summit is available in a recent press release.

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May 2017
Midwest CEPAC

Atopic Dermatitis

Atopic dermatitis (eczema) is a chronic/chronically-relapsing skin condition characterized by itching and dry skin. Atopic dermatitis is common. It affects 5-20% of children worldwide, and approximately 11% of children in the US. It is also estimated to affect around 3-7% of adults in the US. Management of atopic dermatitis can create burdens for the family, and the disorder can decrease quality of life. Itching, in particular, can disrupt sleep and lead to daytime sleepiness, irritability, and psychological stress, and cosmetically important lesions can lead to social stress and isolation.

ICER’s report will evaluate the comparative clinical effectiveness and value of two emerging therapies for atopic dermatitis: crisaborole and dupilumab. Crisaborole will be evaluated based on its expected indication in the treatment of mild-to-moderate atopic dermatitis in children and adults; separately, dupilumab will be evaluated for its expected indication in the treatment of moderate-to-severe atopic dermatitis in adults.

Interventions of Interest:

  • Crisaborole (Eucrisa™, Pfizer, Inc.)
  • Dupilumab (Dupixent®, Sanofi and Regeneron, Inc.)

Date of Review: May 2017

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Mar 2017
New England CEPAC


Rheumatoid arthritis (RA) is the most common autoimmune inflammatory arthritis in adults, affecting approximately 1.5 million Americans. RA is more common in women and may occur at any age, with peak incidence occurring at ages 50-60 years. RA is typically characterized by morning stiffness and symmetrical joint swelling of the feet, hands, and knees, although any joint (and in some cases, internal organs) may be involved. The course of RA may be complicated by cardiac, hematologic, and other extra-articular manifestations.

Interventions of Interest (subject to change):

TNF Inhibitors

  • Adalimumab (Humira®, Abbvie)
  • Certolizumab pegol (Cimzia®, UCB)
  • Etanercept (Enbrel®, Amgen)
  • Golimumab (Simponi®, Janssen)
  • Infliximab (Remicade®, Janssen)

CD20- directed cytolytic antibody

  • Rituximab (Rituxan®, Genentech)

Tcell inhibtor

  • Abatacept (Orencia®, BMS)

IL-6 inhibitors

  • Sarilumab (Kevzara®, Sanofi and Regeneron Pharmaceuticals)
  • Tocilizumab (Actemra®, Genentech)

JAK- inhibitors

  • Tofacitinib (Xeljanz®, Pfizer)
  • Baricitinib (Eli Lilly and Co.)

Date of Review: March 2017


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Feb 2017

Multiple Sclerosis

Multiple sclerosis (MS) is a chronic, immune-mediated inflammatory demyelinating disease of the central nervous system (CNS). Approximately 400,000 Americans have MS, although this may be an underestimate, and the disease affects about twice as many women as men. Some patient groups, such as African Americans, experience a more rapid and severe clinical course. MS is the most common nontraumatic cause of permanent disability among young adults. The annual cost of MS in the United States is estimated to be $28 billion.

ICER’s review will incorporate treatments for two types of MS. The most common form of the disease is relapsing-remitting MS (RRMS), which affects 85% to 90% of patients with MS. RRMS is characterized by periodic relapses characterized by neurologic symptoms that diminish or resolve with treatment. The report will also review treatments for primary-progressive MS (PPMS), which affects approximately 10-15% of MS patients. PPMS is characterized by steadily worsening neurologic function without remissions or acute episodes.

Interventions of Interest:

Injectable Agents

  • Daclizumab (Zinbryta®, Biogen/AbbVie)
  • Glatiramer acetate (Copaxone®, Teva)
  • Glatiramer acetate (Glatopa®, Sandoz (Novartis))
  • Interferon beta-1a (Avonex®, Biogen)
  • Interferon beta-1b (Betaseron®, Bayer)
  • Interferon beta-1b (Extavia®, Novartis)
  • Interferon beta-1a (Rebif®, EMD Serono)
  • Peginterferon beta-1a (Plegridy®, Biogen)

Oral Agents

  • Dimethyl fumarate (Tecfidera®, Biogen)
  • Fingolimod (Gilenya®, Novartis)
  • Teriflunomide (Aubagio®, Sanofi-Genzyme)

Infused Agents

  • Alemtuzumab (Lemtrada®, Sanofi-Genzyme)
  • Natalizumab (Tysabri®, Biogen)
  • Ocrelizumab (Ocrevus®, Roche/Genentech)
  • Rituximab (Rituxan®, Roche/Genentech)

Date of Review: February 2017

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Nov 2016
New England CEPAC


Plaque psoriasis is a common disease that causes red, scaly, raised lesions on the skin, most commonly on the elbows, knees, scalp, and back. Psoriasis affects about 2% of the population and significantly decreases health-related quality of life, particularly if lesions are in areas that can affect daily functioning (e.g., the hands or soles of the feet) or affect social functioning (e.g., the face). Psoriasis is a chronic inflammatory condition that is associated with systemic diseases including psoriatic arthritis, other autoimmune diseases, the metabolic syndrome, and cardiovascular disease.

Interventions of interest:

Anti-TNF agents:

  • Adalimumab (Humira®, AbbVie)
  • Etanercept (Enbrel®, Amgen, Inc.)
  • Infliximab (Remicade®, Janssen)

Anti IL-17A agents:

  • Secukinumab (Cosentyx®, Novartis)
  • Ixekizumab (Taltz®, Eli Lilly and Co.)
  • Brodalumab (SiliqTM,Valeant Pharmaceuticals and AstraZeneca)

Anti IL-12/13 agent:

  • Ustekinumab (Stelara®, Janssen)

Anti-PDE4 agent:

  • Apremilast (Otezla®, Celgene)
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Oct 2016
Midwest CEPAC

Non-Small Cell Lung Cancer

Lung cancer is the number one cause of cancer death in the United States, expected to cause 158,000 deaths in 2016, and accounting for 26.5% of all cancer deaths. It is the second most common cancer in both men (after prostate cancer) and women (after breast cancer), with 118,000 and 106,000 new cases expected in 2016, respectively. Lung cancer includes different pathological types, broadly divided into small-cell lung cancer and non-small-cell lung cancer (NSCLC). NSCLC makes up about 85% of lung cancers and comprises mainly squamous cell carcinoma, adenocarcinoma, and large-cell lung cancer. Stage at diagnosis is a primary factor in patient survival, and patients with NSCLC commonly present with advanced disease.

Interventions of Interest:

Tyrosine kinase inhibitors:
  • Afatinib (Gilotrif®, Boehringer Ingelheim Pharmaceuticals, Inc.)
  • Erlotinib (Tarceva®, Genentech, Inc. and Astellas Pharma US, Inc.)
  • Gefitinib (Iressa®, AstraZeneca)
Immunotherapies targeting the programmed death 1 (PD-1) receptor or its ligand (PD-L1):
  • Atezolizumab (Tecentriq®, Genentech, Inc., Roche Group)
  • Nivolumab (Opdivo®, Bristol-Meyers Squibb Co.)
  • Pembrolizumab (Keytruda®, Merck & Co.)

Date of Review: October 2016

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Jul 2016
New England CEPAC

Obeticholic Acid

Obeticholic acid (OCA) is a novel bile acid analogue that has shown positive effects on biochemical markers of liver function in phase II trials (NCT00550862) and is currently under consideration by the FDA for treatment of primary biliary cholangitis (PBC) after failure of Ursodeoxycholic Acid (UDCA). Primary biliary cholangitis (PBC), which has until recently been called primary biliary cirrhosis, is a rare, chronic, progressive autoimmune liver disease that affects mainly middle-aged women. In the US, up to 130,000 individuals may have PBC.

In January 2015, obeticholic acid also received a breakthrough designation for treatment of non-alcoholic steatohepatitis (NASH) with concomitant liver fibrosis. Interim findings from the phase III trial are not expected to be available until March 2017; however, clinical interest in potential off-label use of obeticholic acid is likely to be great, given the lack of available treatments with liver-specific effects. NASH is form of nonalcoholic fatty liver disease (NAFLD) that can progress to cirrhosis, liver failure, and cancer. NASH is present in about 30 million adults in the US.

Intervention of Interest: Obeticholic Acid (Intercept Pharmaceuticals, Inc.)

Date of Review: July 2016

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